On December 17, 2020 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that the US Food and Drug Administration (FDA) is allowing the company’s Investigational New Drug (IND) application to study Annamycin for the treatment of soft tissue sarcoma lung metastases to go into effect (Press release, Moleculin, DEC 17, 2020, View Source [SID1234572975]). This allows Moleculin to begin a Phase 1B/2 clinical trial in the US for patients with soft tissue sarcoma that has metastasized to the lungs after first-line therapy for their disease.
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It is estimated that there are approximately 36,000 new cases of soft tissue sarcoma (STS) in the 7 major markets (US, EU5 and Japan) each year, and an estimated annual market size of over $177 million for the treatment of STS lung metastases. Our clinical advisors estimate that approximately half of all STS patients will eventually develop lung metastases from their primary tumor. Although first-line treatments such as surgical resection, chemotherapy and radiation may provide initial therapeutic benefit for an estimated 35% of those patients, there are no approved or emerging second-line therapies for the remaining 65% who relapse or are refractory. Although the lungs tend to be a major site of relapse, we are aware of only 2 active clinical trials specifically targeting STS lung metastases, indicating that Annamycin faces limited competition in this area of development.
Moleculin recently announced that Annamycin demonstrates consistently high antitumor activity in vivo in all tested animal models of different types of lung-localized cancers, including sarcoma. These promising findings correlate with surprisingly high uptake of Annamycin to the lungs in animal models. This uptake is up to 34-fold higher than that of doxorubicin, the primary first-line chemotherapy for STS. The limited pulmonary uptake of doxorubicin in animal models may help explain its lack of activity against STS lung metastases in humans. Additionally, clinical data show no cardiotoxicity associated with the use of Annamycin, as well as the ability to avoid multidrug resistance mechanisms, both of which are often treatment-limiting effects of anthracyclines (which includes doxorubicin) in this setting. Taken together, these factors suggest that Annamycin could represent an important treatment to help address a significant unmet need in patients with STS lung metastases.
"Since the discovery in animal models of Annamycin’s effectiveness in lung metastases, we have been moving quickly to begin a clinical trial in the US to study Annamycin for this indication," commented Walter Klemp, Chairman and CEO of Moleculin. "Recognizing the importance of building on the results with human clinical data, we preemptively included this upcoming trial as part of our budget plan, so our anticipated cash runway into the third quarter of 2021 remains unchanged. We are also collaborating with our partners and physicians in Poland who have shown a high level of interest in testing Annamycin in STS lung metastases and are currently pursuing a possible investigator-led clinical trial in Europe. It is our goal to have to have our US clinical trial begin by mid-2021, with the possibility of also initiating a European investigator-funded clinical trial in 2021."
Mr. Klemp concluded: "Along with the results in STS lung metastases, our animal models have shown significant activity in other lung metastases, including colorectal and triple negative breast cancer, as well as meaningful concentration levels of Annamycin in the liver, spleen and pancreas. Additionally, when tested in a highly aggressive AML mouse model, Annamycin significantly reduced tumor burden in the spleen, lungs and liver, leading to a significant increase in survival. Based on this promising preclinical data, we believe the ultimate market opportunity for Annamycin could be much larger than just STS lung metastases. For all these reasons, we are excited to be moving from the animal models to clinical study."